There may be drugs at the industry's fingertips, already approved, that could be of value in treating patients with neurodegenerative disease if repurposed and brought to regulatory approval for expanded indications, but these may be stalled due to rundown patents and a lack of financial incentive in the market, according the a study published June 24 in the Annals of Clinical and Translational Neurology.
Diana Shineman, PhD, director of scientific affairs for the Alzheimer's Drug Discovery Foundation in New York City, and colleagues discussed the challenges and provided a potential multi-stakeholder game plan between governmental agencies and non-governmental organizations. Contributing authors came from all sides of the fence, including Mt. Sinai in New York City, the Cleveland Clinic in Las Vegas, Sunnybrook Health Sciences at the University of Ontario, Northwestern University in Chicago, the Michael J. Fox Foundation, and AstraZeneca based in Waltham, Mass.
"Through utilization of large data sets, electronic medical records, and bioinformatics, we can identify new unexpected opportunities for drug repurposing," wrote the authors. "However, those opportunities require funding to prove that the drug will be safe and effective in the target patient population."
The remedy to the challenging market landscape presented in the study involves building momentum for groups already funding trials for repurposed drugs. Some of these groups include the Cures Within Reach foundation, the Alzheimer's Society (UK), Cure Parkinson's Trust and the Multiple Myeloma Research Foundation. Organizations such as the Leukemia Lymphoma Society and the National Center for Advancing Translational Sciences at the National Institutes of Health are also working together to bring possibilities to a reality. One example of a repurposed drug is the repurposing of Auranofin, originally prescribed for chronic lymphocytic leukemia. Thalidomide and Methotrexate are other examples of repurposed drugs for neurodegenerative disease.
The Alzheimer' Disease Cooperative Study could also spur more interest in repurposing agents and distributing through generic companies. Funding needs to come from not just research and development for new agents, but government grants and public-private organizations to get more trials on the books and more data in the literature. Amassing consortiums between all interested parties might provide the right atmosphere to support repurposing of approved drugs.
"In the current climate, the funding is not there and repurposed drugs are unlikely to reach the patient because companies typically have insufficient financial incentive," wrote Shineman et al. "Innovations in policy, risk sharing, and collaborations may change that reality. Foundations and government can drive the change by investing in the necessary proof-of-concept efficacy studies and advocating for policy changes that will incentivize industry. Through the strategy options outlined above, we can create feasible commercial paths to test promising repurposed agents for neurodegenerative disease and accelerate the availability of drugs to the patients in need.